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1. (WO2019009298) α-SYNUCLEIN EXPRESSION INHIBITOR
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Pub. No.: WO/2019/009298 International Application No.: PCT/JP2018/025236
Publication Date: 10.01.2019 International Filing Date: 03.07.2018
IPC:
C12N 15/113 (2010.01) ,A61K 31/712 (2006.01) ,A61P 25/16 (2006.01) ,A61P 25/28 (2006.01)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
712
Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25
Drugs for disorders of the nervous system
14
for treating abnormal movements, e.g. chorea, dyskinesia
16
Anti-Parkinson drugs
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25
Drugs for disorders of the nervous system
28
for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
Applicants:
国立大学法人大阪大学 OSAKA UNIVERSITY [JP/JP]; 大阪府吹田市山田丘1番1号 1-1, Yamadaoka, Suita-shi, Osaka 5650871, JP
国立研究開発法人医薬基盤・健康・栄養研究所 NATIONAL INSTITUTES OF BIOMEDICAL INNOVATION, HEALTH AND NUTRITION [JP/JP]; 大阪府茨木市彩都あさぎ七丁目6番8号 7-6-8, Saito-Asagi, Ibaraki-shi, Osaka 5670085, JP
第一三共株式会社 DAIICHI SANKYO COMPANY, LIMITED [JP/JP]; 東京都中央区日本橋本町三丁目5番1号 3-5-1, Nihonbashi Honcho, Chuo-ku, Tokyo 1038426, JP
Inventors:
中森 雅之 NAKAMORI, Masayuki; JP
望月 秀樹 MOCHIZUKI, Hideki; JP
小比賀 聡 OBIKA, Satoshi; JP
笠原 勇矢 KASAHARA, Yuya; JP
小泉 誠 KOIZUMI, Makoto; JP
中村 晃史 NAKAMURA, Akifumi; JP
高石 巨澄 TAKAISHI, Kiyosumi; JP
朝日 結実子 ASAHI, Yumiko; JP
Agent:
植木 久一 UEKI, Kyuichi; JP
植木 久彦 UEKI, Hisahiko; JP
菅河 忠志 SUGAWA, Tadashi; JP
伊藤 浩彰 ITO, Hiroaki; JP
Priority Data:
2017-13228805.07.2017JP
Title (EN) α-SYNUCLEIN EXPRESSION INHIBITOR
(FR) INHIBITEUR DE L'EXPRESSION DE L'α-SYNUCLÉINE
(JA) α-シヌクレイン発現抑制剤
Abstract:
(EN) According to the present invention, a nucleic acid medicine, which has a higher effect of inhibiting α-synuclein expression and sustains this effect for a longer period of time, can be provided. The oligonucleotide according to the present invention or a pharmacologically acceptable salt thereof is characterized by: containing at least one nucleoside structure represented by formula (I) [wherein each of "Base" and "A" represents a definite substituent or structure]; being capable of binding to an α-synuclein gene; having an activity of inhibiting the expression of the α-synuclein gene; being complementary to the α-synuclein gene; having a specific sequence; being complementary to at least a part of α-synuclein represented by SEQ ID NO: 1; and having an oligonucleotide length of 12-20 bases.
(FR) La présente invention concerne un médicament à base d'acide nucléique, qui présente un effet d'inhibition de l'expression de l'α-synucléine plus élevé et qui maintient cet effet pendant une durée plus longue. L'oligonucléotide selon la présente invention ou un sel pharmacologiquement acceptable de ce dernier est caractérisé : en ce qu'il contient au moins une structure nucléosidique représentée par la formule (I) [dans laquelle « Base » et « A » représentent chacun un substituant défini ou une structure définie] ; en ce qu'il est capable de se lier à un gène d'α-synucléine ; en ce qu'il présente une activité d'inhibition de l'expression du gène de l'α-synucléine ; en ce qu'il est complémentaire du gène de l'α-synucléine ; en ce qu'il présente une séquence spécifique ; en ce qu'il est complémentaire d'au moins une partie de l'α-synucléine représentée par SEQ ID NO : 1 ; et en ce qu'il présente une longueur d'oligonucléotide de 12 à 20 bases.
(JA) 本発明によれば、α-シヌクレインの発現抑制について、より高い効果およびより長い持続性を有する核酸医薬を提供することができる。本発明に係るオリゴヌクレオチドまたはその薬理学上許容される塩は、以下の式(I)で表されるヌクレオシド構造:(式中BaseおよびAは所定の置換基または構造である)を少なくとも1つ含み、α-シヌクレイン遺伝子と結合することができ、該α-シヌクレイン遺伝子の発現を抑制する性を有し、そして該α-シヌクレイン遺伝子と相補的であり、特定の配列を有し、配列番号1のα-シヌクレインの少なくとも一部と相補的で、そして該オリゴヌクレオチドの長さが、12~20塩基であることを特徴とする。
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Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: Japanese (JA)
Filing Language: Japanese (JA)